- A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG — Active Not Recruiting • Phase II / Phase III • NCT06892288.
- Weekly GLM101 treatment tested against placebo for a rare genetic disorder affecting protein processing in children and adults.
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This study is evaluating the safety, effectiveness, and how the body absorbs, distributes, and eliminates GLM101, for participants with PMM2-CDG, including children, adolescents, and adults. Researchers will compare participants receiving GLM101 to those receiving a placebo to see if GLM101 improves symptoms of PMM2-CDG. The study includes two treatment parts: a 24-week double blind placebo-controlled treatment period (Part A), and a 24-week open-label phase where every participant will receive GLM101(Part B). Conditions: Phosphomannomutase 2 Deficiency, PMM2-CDG Interventions: GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label) Lead…